• 11:30 AMTrial architecture for small, heterogeneous populations — lessons from NPC, sphingolipidoses, and rare neurodegenerative LSDsRaphael Schiffmann, MD
• 11:50 AMAdaptive, pragmatic, and decision-focused trial designsTerrence Eagleton · Medpace, Ohio, USA
• 12:10 PMUse of composite endpoints in Gaucher disease and other rare disordersAri Zimran, MD · Shaare Zedek Medical Center, Jerusalem, Israel
• 12:30 PMSession Q&A — Open discussion with all session speakers

Measuring meaningful benefit beyond biochemical correction

• 1:30 PMAppropriate use of natural history data as external controls: when it works and when it failsDavid Kronn, MD · New York Medical College, New York, USA
• 1:50 PMEndpoint selection in pediatric-onset lysosomal disorders and prediction of adult outcomesUma Ramaswami, MD · Royal Free Hospital / UCL, London
• 2:10 PMPatient-centered outcomes and trial burden in lysosomal disordersTanya Collin-Histed · CEO, International Gaucher Alliance, UK
• 2:30 PMSession Q&A — Open discussion with all session speakers

Biomarkers as decision-support tools; distinguishing disease progression from treatment-related risk

• 3:15 PMPharmacodynamic vs. disease-burden biomarkers: Fabry disease as the model caseBehzad Najafian, MD, PhD · University of Washington, Washington, USA
• 3:35 PMRenal biomarker harmonization and longitudinal interpretationDavid Warnock, MD · University of Alabama at Birmingham, Alabama, USA
• 3:55 PMBiomarkers for patient selection and enrichment in NPC trials: oxysterols, NfL, and cholestane-triolCaroline Hastings, MD · University of California, San Francisco, USA
• 4:15 PMInterpreting biomarkers in treated and late-treated patients: when numbers misleadNeal Weinreb, MD · University of Miami / Research Foundation for LSDs, Florida, USA
• 4:35 PMSession Q&A — Open discussion with all session speakers

Partial responders, CNS vs. peripheral benefit, and durability

• 9:00 AMThe clinical trial endpoint journey, and lessons learned on how to design a trial for approval for small moleculesDominique Germain, MD, PhD · University of Versailles / Paris-Saclay University, France
• 9:20 AMLessons from GBA1-associated Parkinson disease trialsAndres Deik, MD · University of Pennsylvania (UPenn), Pennsylvania, USA
• 9:40 AMNPC as the case study: NALL, the NPCCSS endpoint journey, and lessons from a CRL to first FDA approvalFrances Platt, MD · University of Oxford, UK

Immunogenicity, residual disease, and real-world effectiveness

• 10:00 AMResidual disease in treated Fabry patients and implications for future trialsDerralynn Hughes, MD, PhD · Royal Free Hospital / UCL, London
• 10:20 AMNeuronopathic lysosomal disorders in the treated era: biomarkers, neurocognition, and residual diseaseJoseph Muenzer, MD, PhD · University of North Carolina, North Carolina, USA
• 10:40 AMChallenges in global clinical trial execution for sphingolipidosesFatih Ezgu, MD · Gazi University, Ankara, Turkey
• 11:00 AMSession Q&A — Open discussion with all session speakers

Clinical management after dosing

• 11:45 AMDurability of response and loss of efficacy following gene therapyArian Pano, MD · Industry Gene Therapy Programs
• 12:05 PMWhen stabilization equals success: endpoints in neurodegenerative lysosomal disordersRoberto Giugliani, MD, PhD · Federal University of Rio Grande do Sul, Porto Alegre, Brazil
• 12:25 PMPost-trial monitoring and real-world implementation following gene therapyTahsin Muzaffar, MD · Industry Advanced Therapy Programs
• 12:45 PMImmune risk stratification and patient eligibility criteria for gene therapy trialsCoy Heldermon, MD, PhD · Professor of Hematology & Oncology, University of Florida / Shands Cancer Center, Florida, USA
• 1:05 PMSession Q&A — Open discussion with all session speakers
• 1:25 PMClosing Remarks & Adjournment — Program Chair